Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy

By Sevin Caroline   Benraiss Abdellatif   Van Dam Debby   Bonnin Delphine   Nagels Guy   Verot Lucie   Laurendeau Ingrid   Vidaud Michel   Gieselmann Volkmar   Vanier Marie   De Deyn Peter Paul   Aubourg Patrick   Cartier Nathalie  

Human Molecular Genetics, Vol. 15, Iss. 1, 2006-01 ,pp. : 53-64

Oxford University Press

Access to resources Recommend Favorite

Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age

By Matthes Frank   Stroobants Stijn   Gerlach Debora   Wohlenberg Claudia   Wessig Carsten   Fogh Jens   Gieselmann Volkmar   Eckhardt Matthias   D'Hooge Rudi   Matzner Ulrich  

Human Molecular Genetics, Vol. 21, Iss. 11, 2012-06 ,pp. : 2599-2609

Oxford University Press

Access to resources Recommend Favorite

Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy

By Matzner Ulrich  

Human Molecular Genetics, Vol. 14, Iss. 9, 2005-05 ,pp. : 1139-1152

Oxford University Press

Access to resources Recommend Favorite

Targeted Disruption of the Mouse Sphingolipid Activator Protein Gene: A Complex Phenotype, Including Severe Leukodystrophy and Wide-Spread Storage of Multiple Sphingolipids

By Fujita Nobuya   Suzuki Kinuko   Vanier Marie T.   Popko Brian   Maeda Nobuyo   Klein Andreas   Henseler Margarete   Sandhoff Konrad   Nakayasu Hiroyuki   Suzuki Kunihiko  

Human Molecular Genetics, Vol. 5, Iss. 6, 1996-01 ,pp. : 711-725

Oxford University Press

Access to resources Recommend Favorite

The expanding phenotype of RNU4ATAC pathogenic variants to Lowry Wood syndrome

By  

American Journal Of Medical Genetics Part A, Vol. 176, Iss. 2, 2018-02 ,pp. : 465-469

John Wiley & Sons Inc

Access to resources Recommend Favorite